Scholar Rock (SRRK) Piper Sandler 36th Annual Healthcare Conference summary

Key clinical data and patient impact

• Phase III Sapphire study in SMA met its primary endpoint, showing a 1.8-point gain on the Hammersmith scale, which is clinically meaningful and represents a significant improvement over standard of care alone.

• 30% of patients achieved a three-point or greater increase, with consistent benefits across all subgroups.

• Apitegromab was well tolerated with a clean safety profile, supporting chronic use and potential as a new standard of care.

• The therapy addresses the community's need for functional gains and maintenance, with strong advocacy group support and high patient anticipation.

• Monthly IV administration is not seen as a barrier, with high long-term adherence and plans for home infusion options.

Commercial outlook and launch readiness

• Market opportunity is estimated at over $1 billion, with potential to expand beyond SMA into adjacent indications.

• The product is positioned as additive to existing SMN therapies, not as a competitor, enhancing the overall treatment landscape.

• Launch preparations are well underway, including regulatory submission targeted for Q1, strategic commercial hires, and payer engagement.

• High completion and rollover rates from clinical trials indicate strong patient and physician buy-in.

• Momentum is building for a commercial launch in 2025, with supply chain and market access strategies in place.

Pipeline and future development

• EMBRAZE phase II trial is evaluating apitegromab plus GLP-1 for obesity, aiming to preserve lean mass rather than increase weight loss.

• The approach targets myostatin selectively, aiming to avoid toxicity and improve metabolic outcomes.

• Success in EMBRAZE is defined by preservation of lean mass, with additional endpoints including A1C and body composition.

• Regulatory endpoints in obesity may evolve, but current focus is on demonstrating added clinical benefit and safety.

• Plans are in place to expand into cardiometabolic and neuromuscular adjacencies, leveraging the platform's selectivity and safety.